What Will 2011 Hold for CER and PCORI?

When future historians consider health care, they may well look back on 2010 as a watershed year in America. The issues and events that occurred last year, most notably the passage of the Patient Protection and Affordable Care Act (PPACA), represent a significant evolution and set the foundation for a profound change in how health care is practiced and managed in this country.  With these significant changes to our system still fresh in our minds, it is a good time to take a few moments to reflect on the important issues and activities of the past year and consider how these events will continue to evolve and play out in the coming year and beyond. 

A significant element of the PPACA builds on the 2009 passage of the American Recovery and Reinvestment Act (ARRA) that provided $1.1 billion1 in stimulus funds to invest in research directed at improving health care for Americans. PPACA reinforced this commitment to improved health with its support of enhanced knowledge about the effectiveness of the full range of medical treatments and understanding of which treatments work best and for whom and under what circumstances. Comparative effectiveness research (CER) is intended to enable this new level of knowledge and understanding, and CER activities intensified during 2010 and will continue to do so in 2011.2

Awareness and knowledge grew in 2010 about the broad spectrum of activities that CER encompasses: analysis of current evidence, conduct of new studies, and dissemination of the information to influence care decisions. Numerous projects received funds from the National Institutes of Health (NIH), the Agency for Health Care Research and Quality (AHRQ), and the Health and Human Services (HHS) Office of the Secretary, the agencies charged with disbursing or applying the ARRA funds for CER. These projects provided practical realization of the CER spectrum of issues and the activities needed to address them. While NIH was quick to begin obligating its $400 million in ARRA funds in 2009, nearly all of the $700 million provided to AHRQ and HHS was obligated in 2010.

The new health care reform law ended the short life of the oversight committee for ARRA CER funds – the Federal Coordinating Council for CER-- but the law required the creation of a new non-profit Patient Centered Outcomes Research Institute (PCORI) to guide both the conduct of CER research and the dissemination of CER findings. PPACA provides new and increased funding for CER that will reach an annual total of $600 million.3   Appointed in September 2010, the members of the Board of Governors for PCORI—a well-qualified and diverse group of 21 health care experts, including the directors of AHRQ and NIH—met officially for the first time in November and again in January. Among the key challenges faced by PCORI is establishing the methodological framework that will help to define high-quality CER. Although comparative effectiveness research is not new, significant methodological questions are well-known but unanswered. The health care reform law provides appropriate recognition of the issue by calling for the establishment of a methods committee as part of PCORI.  Nominations for this committee are under consideration, and the new members will be announced in the near future.

The PCORI’s work is beginning in earnest this year, and one of its initial and essential tasks is to establish the priorities for CER that will guide its future activities. While others, including the Institute of Medicine (IOM), have offered their recommended priorities for research4, the PCORI has the responsibility not only for establishing the priorities, but also for acting upon them and being accountable for the research activities that are funded to address those priorities.5   The process for establishing priorities must be well-structured and have the benefit of input from the full range of stakeholders, including patients, providers, and suppliers, that will be impacted by the application of findings that result from CER.  According to the law, the methodology committee has 18 months from the establishment of PCORI to develop the specifications for appropriate CER methods and must provide a “translation table” that will provide guidance to the PCORI Board in matching research questions to the appropriate methods that will best address each question.  These activities will provide essential guidance as well to the researchers that will conduct the research.6

Another issue to be addressed is the process for selecting researchers that will carry out the CER research agenda. While PCORI is a non-profit corporation, the PPACA law nevertheless gives preference in contracting for the research to AHRQ and NIH, the two Federal agencies that have to-date conducted the lion’s share of federally supported CER. The heads of those two agencies have seats on the PCORI Board of Governors.   But there are several private sector groups that currently support the CER needs of private payers and some public payers. The PPACA law suggests opportunities for these private sector groups, as well as academic researchers, but how important will their role be in the new CER environment? What criteria will be used to determine when opportunities will be afforded to these non-Federal researchers?

As the PCORI discussions evolve, there will be significant interest in how effectiveness is defined and addressed in priorities and research methodology. The important questions about what treatments or means of providing care work best must have a “real world” context, i.e., the clinical settings in which patients receive care. Thus, there will be important methodological issues to be addressed regarding the extent to which data generated in patient care – observational data – can be relied upon to provide answers to the comparative effectiveness questions that will be defined and prioritized by the PCORI Board. Outside of PCORI, a recently developed set of principles, the Good ReseArch for Comparative Effectiveness (GRACE) Principles, establishes a useful standard for the quality of CER using observational data, including references to specific examples in which observational research has been particularly helpful.7  However, there is also considerable debate and there are strongly held views on randomized controlled studies as the basis on which treatment effectiveness questions must be ultimately resolved.8

The IOM has noted frequently the need for a “learning” health care system9 , and if health care in the U.S. is to learn from experience, how can data that describes experience in the clinical setting be used to provide sound and reliable learning? There are already numerous databases of information that describe the care provided to individual patients, and efforts are ongoing to find ways of sharing that data to address some of the gaps in research evidence. New requirements, in part as a result of PPACA, to create computerized medical records and increase availability of those records through health information technology systems will reinforce the need to resolve questions about how best to use these data resources. The PCORI Board and the methodology committee, with appropriate counsel from external experts, will have to play a key role in guiding researchers on these issues and building confidence in the methods that are recommended as a result.

Clearly, there are substantive issues related to the generation of new comparative effectiveness research, but there are equally important questions about how the evidence from this research gets disseminated and translated to clinical use. PPACA has specific requirements for PCORI to release publicly the findings from the CER it supports, and AHRQ is charged with broadly disseminating research findings to key stakeholders.  What are the most efficient and effective ways of translating and providing this evidence to providers and patients?  What is needed to ensure that decisions about benefit designs adequately and accurately take account of CER findings? How will the findings from CER affect the measures that are used to track and improve the quality of care? The health care reform law includes opportunities to implement and test some new ways of organizing and providing patient access to care, notably, Accountable Care Organizations among others.10 How will the evidence generated through CER affect the accountability of these new health care system experiments? It is likely that few specific answers will be provided to these questions during 2011, but it will be an important time to give them thoughtful consideration as new CER begins.

As 2011 gets underway, it is also helpful to remember that while scientific and technical issues may be paramount in this new CER world, the PCORI and its responsibilities were created through a political process. This new year begins with a significant change in politics at the Federal level and in many instances at the state level where there is strong opposition to the health care reform law.  Aside from complete repeal, there is little indication regarding what this general opposition to the reform law may mean for CER specifically. So as historians consider and chronicle the monumental health care changes of 2010, they know full–well the final chapter has not yet been written.

1 111th Congress of the United States. American Recovery and Reinvestment Act of 2009.H.R. 1. January 6, 2009. Washington, D.C.: U.S. Government Printing Office; http://frwebgate.access.gpo.gov/cgi-bin/getdoc.cgi?dbname=111_cong_bills&docid=f:h1enr.pdf. Accessed January 3, 2011.

2 Department of Health and Human Services. American Recovery and Reinvestment Act. Supporting Comparative Effectiveness Research. National Institutes of Health: Comparative Effectiveness Research. http://www.hhs.gov/recovery/reports/plans/pdf20100610/NIH_Comparative%20Effectiveness%20%20June%202010.pdf. Accessed January 3, 2011; Department of Health and Human Services. American Recovery and Reinvestment Act. Agency for Healthcare Research and Quality: Comparative Effectiveness Research. http://www.hhs.gov/recovery/reports/plans/pdf20100610/AHRQ%20CER%20June%202010.pdf. Accessed January 3, 2011. Department of Health and Human Services American Recovery and Reinvestment Act. Funds for Allocation by the Office of the Secretary through the Agency for Healthcare Research and Quality (AHRQ): Comparative Effectiveness Research. http://www.hhs.gov/recovery/reports/plans/pdf20100610/OS%20CER%20June%202010.pdf. Accessed January 3, 2011.

3 Clancy C, Collins FS. Patient-centered outcomes research institute: the intersection of science and health care. SciTransl Med. 2010;2:37cm18.

4 Institute of Medicine. Initial National Priorities for Comparative Effectiveness Research. Washington, DC: National Academies Pr; 2009. Accessed at http://www.nap.edu/catalog.php?record_id=12648 on January 3, 2011.

5 Patient Protection and Affordable Care Act, p. 618.

6 Patient Protection and Affordable Care Act, p. 615.

7 Good ReseArch for Comparative Effectiveness. http://www.graceprinciples.org/. Accessed on January 3, 2011.

8 NIH/AHRQ Conference on Methodological Challenges in Comparative Effectiveness Research. http://videocast.nih.gov/Summary.asp?File=16313. Accessed on January 3, 2011

9 Institute of Medicine.The Learning Healthcare System in 2010 and Beyond: Understanding, engaging, and communicating the possibilities. http://www.iom.edu/Activities/Quality/VSRT/2010-APR-01.aspx. Accessed January 3, 2011

10 Patient Protection and Affordable Care Act, p. 277, p.821.